1. Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment. Issue 11 (29th July 2017) Authors: Mauer, Michael; Sokolovskiy, Alexey; Barth, Jay A; Castelli, Jeffrey P; Williams, Hadis N; Benjamin, Elfrida R; Najafian, Behzad Journal: Journal of medical genetics Issue: Volume 54:Issue 11(2017) Page Start: 781 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. Issue 4 (10th November 2016) Authors: Hughes, Derralynn A; Nicholls, Kathleen; Shankar, Suma P; Sunder-Plassmann, Gere; Koeller, David; Nedd, Khan; Vockley, Gerard; Hamazaki, Takashi; Lachmann, Robin; Ohashi, Toya; Olivotto, Iacopo; Sakai, Norio; Deegan, Patrick; Dimmock, David; Eyskens, François; Germain, Dominique P; Goker-Alpan, O... Journal: Journal of medical genetics Issue: Volume 54:Issue 4(2017) Page Start: 288 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗